Pfizer's Tafamidis Nominated for Priority Review in China for Rare TTR Amyloidosis Indications

On February 3, the CDE website announced that Pfizer’s tafamidis soft capsules have been included in the proposed priority review program under Category (II) of the priority review scope for four rare diseases.

Tafamidis is a transthyretin (TTR) stabilizer. Currently, its approved indications include hereditary transthyretin-mediated amyloid polyneuropathy and transthyretin amyloid cardiomyopathy. The drug was initially approved for marketing in the European Union on November 15, 2011, received FDA approval on May 7, 2019, and Pfizer’s marketing application for tafamidis was accepted by the National Medical Products Administration of China on July 26, 2019.

Transthyretin Familial Amyloid Polyneuropathy is a rare disease, an autosomal dominant amyloidosis caused by gene mutations. Currently, there are no effective treatments available. Previously, this drug was included in China’s “List of the First Batch of Clinically Urgent Overseas New Drugs.”

Tafamidis is available in two oral formulations, marketed under the brand names Vyndaqel and Vyndamax. Pfizer’s 2019 annual report indicated that the drug generated $473 million in sales in 2019. Analysts predict that its peak annual sales will exceed $1.5 billion.